Alexion Pharmaceuticals, Inc., headquartered in Boston, Massachusetts in the United States, is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing medicines.

With the development of its first therapy, Alexion emerged as the global leader in complement science, with more than 20 years of experience in complement research and discovery. It has since developed and delivered therapies for the treatment of four complement-mediated diseases – two for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), and one for patients with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD) and anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG). It also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders—hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). Alexion has received some of the pharmaceutical industry's highest honors: the Prix Galien USA (2008, Best Biotechnology Product), France (2009, Rare Disease Treatment) and Germany (2016, Orphan Product).

In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases as well as several early-stage therapies, including one for light chain (AL) amyloidosis and a second anti-FcRn therapy.

The company’s research efforts are focused on novel molecules and targets in the complement cascade. Its development programs are focused on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Alexion views it’s R&D efforts are crucial to delivering life-changing medicines to patients and families affected by rare diseases who often have few, if any, effective treatment options. At the same time, the company is leveraging a proprietary bioinformatics platform to strengthen its understanding of rare diseases and provide insights into their epidemiology.

By continuing to deepen its understanding of rare disease, Alexion is able to innovate and evolve into new areas, where there is great unmet need and opportunity to help patients and families fully live their best lives. It is this understanding that fuels all of its efforts, beginning with its medicine discovery efforts, as well as its collaboration with external partners. In April 2018, Alexion expanded its metabolics portfolio with the acquisition of Wilson Therapeutics. More recently, in October 2019, the business announced its intent to acquire Achillion Pharmaceuticals, further building upon its nearly three decades of expertise in complement biology. As a fully-integrated biopharmaceutical company, Alexion brings world-class expertise and resources to its collaborations, including proven global capabilities spanning scientific, medical, regulatory, manufacturing and commercial operations.

Alexion also partners with patient advocacy organizations and actively listens to patient communities in order to better understand and deliver the support they need.

Alexion has more than 3,000 employees around the world and serves patients in more than 50 countries. Its culture is rooted in integrity, inclusiveness, and dedication to joining and supporting its communities. At Alexion, passion drives continuous innovation and creates meaningful value in everything that it does.


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